Medical and Regulatory Affairs

Clinical Research: Phase 1 - Phase 4

PREMIER VOICES #14: Setting a Real-World Strategy in an Evolving Clinical Research Environment

Featured Guests Stacy WeilSenior Vice President,Clinical Data Operations, Strategic Business Optimization Stacy Weil translates Premier Research’s strategy into actionable data initiatives that enable internal and external customers to achieve the highest level of performance and success. Prior to joining Premier Research, Ms. Weil was Vice President of Clinical Data Operations at PatientiP.Read More Nach Davé,...

Medical and Regulatory Affairs

The FDA Is Ending Its Rare Pediatric Disease Priority Review Vouchers – and Time Is Running Out to Get One

Between 2010 and 2018, 33.8 percent of the 402 orphan indications approved by the FDA were approved for children only or targeted pediatric diseases.[1] The rare pediatric disease priority review voucher program is one incentive that’s helped to spur research and development in this area with more than 20 vouchers granted to date, but it...

Clinical Research: Phase 1 - Phase 4

PREMIER VOICES #13: Early-Phase Oncology – Emerging From the Crisis & Innovating for the Future

Featured Guests Luke Gill, B.Sc., M.Sc., MBAVice President, Oncology Luke Gill is the head of Oncology Clinical Development Services at Premier Research. He works collaboratively with biotech sponsors, the scientific community, and other stakeholders to develop and implement strategic approaches to early phase oncology development and CAGT trials. As a drug development expert, Gill has...

Consulting

Creating a Compliant eCTD: Avoiding Common Regulatory and Technical Pitfalls on the Path to Submission

The Electronic Common Technical Document (eCTD) is the standard format for submitting applications, amendments, supplements, and reports to the U.S. Food & Drug Administration (FDA). The eCTD harmonizes the regulatory review process for global drug development, as its structure is based on the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use...

Consulting

Long-Term Follow-Up in Gene Therapy Trials: Ensuring Patient Engagement & Regulatory Compliance

Gene therapy products are typically designed to achieve therapeutic effect through long-acting or permanent changes in the human body. Due to this extended period of exposure, patients who receive gene therapies may be at increased risk of delayed adverse events. To understand and mitigate the risk of these delayed adverse events, participants in gene therapy...

Consulting

Why ePROs Are Quickly Becoming a Key Source for Greater Patient Engagement

With the need to incorporate remote ways of working and interacting with study participants, sponsors and contract research organizations (CROs) have turned to technology for critical study activities ranging from patient consent and consultation to site monitoring. A particular instance of increased technology uptake has been in the area of patient-reported outcome (PRO) data collected...

Medical and Regulatory Affairs

Asia-Pacific – Taking Device Clinical Development to the USA and Europe: Understanding the Changes & Leveraging the Opportunity

Medical devices play an increasingly critical role in the health and quality of life for millions of people worldwide. While clinical trials for medical devices have many similarities to those for pharmaceuticals, the regulatory evaluation of devices is distinct from that of drugs – and there are critical differences in the way the device trials...

Medical and Regulatory Affairs

iPSP Considerations for Molecularly Targeted Cancer Drugs

With the passage of the Pediatric Research Equity Act (PREA) of 2003, drug manufacturers were required to submit an initial pediatric study plan (iPSP) prior to commencement of Phase 3 studies (or new drug application [NDA]/biologics license application [BLA] submission in the absence of a Phase 3 study) for studies involving a new active ingredient,...

Medical and Regulatory Affairs

5 Things to Know About the FDA RACE for Children Act

The Research to Accelerate Cures and Equity (RACE) for Children Act aims to improve and expand treatment options for pediatric cancer patients by mandating that all new adult oncology drugs also be tested in children when the molecular targets are relevant to a particular childhood cancer. Enacted August 18, 2017, as part of the Food...

Consulting

Snapshot: A New Guidance Document Program From the FDA

As a regulatory affairs professional advising various stakeholders on the interpretation and implementation of guidance documents from the FDA, I can safely say that the task is challenging in the best of cases. FDA guidance documents have a tendency to be vague and difficult to decode. As experts, we are always debating about what the...